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LV1.        Characterization of VIRxSYS lentiviral vector HIV vaccine candidate

Lemiale F, Asefa B, Ye D, Chen C, Korokhov N, Humeau L.

Vaccine. 2010 Feb 23;28(8):1952-61

 

LV2.        Heterologous vaccination protocol with lentiviral vectors

Asefa B, Korokhov N, Lemiale F.

Vaccine. 2010 Apr 30;28(20):3617-24

 

LV3.        Collaborative work with University of California Berkeley related to VRX496 MOA

Willerth SM, Pedro HA, Pachter L, Humeau LM, Arkin AP, Schaffer DV.

PLoS One. 2010 Oct 22;5(10):e13564.

 

LV4.        A Review of lentiviral vectors used as vaccine

Lemiale F, Korokhov N.

Vaccine. 2009 May 26;27(25-26):3443-9

 

LV5.        A Review of therapeutic approaches using lentiviral vectors in the clinic

D'Costa J, Mansfield SG, Humeau LM.

Curr Opin Mol Ther. 2009 Oct;11(5):554-64

 

LV6.        Collaborative work with University of Pennsylvania related to CD4 T cell transduction

Agosto LM, Yu JJ, Liszewski MK, Baytop C, Korokhov N, Humeau LM, O'Doherty U.

J Virol. 2009 Aug;83(16):8153-62

 

LV7.        Collaborative work with University of Pennsylvania related to ex-vivo DC based vaccine approach

Wingard JB, Anderson B, Weissman D.

Eur J Immunol. 2008 May;38(5):1310-20

 

LV8.        Collaborative work with University of Pennsylvania related to CD4 T cell transduction and HIV infection

Richardson MW, Carroll RG, Stremlau M, Korokhov N, Humeau LM, Silvestri G, Sodroski J, Riley JL.

J Virol. 2008 Nov;82(22):11117-28

 

LV9.        Collaborative work with Harvard Medical School related to transduction and protection against SIV infection

Braun SE, Lu XV, Wong FE, Connole M, Qiu G, Chen Z, Slepushkina T, Slepushkin V, Humeau LM, Dropulic B, Johnson RP.

Hum Gene Ther. 2007 Jul;18(7):653-64

LV10.     Phase I Clinical Trial
Gene Transfer in Humans using a Conditionally Replicating Lentiviral Vector
Levine B, Humeau L, Boyer J, MacGregor RR, Rebello T, Lu X, Binder GK, Slepushkin V, Lemiale F, Mascola J, Bushman F, Dropulic B, and June C. Gene Transfer in Humans using a conditionally replicating Lentiviral Vector. Proceedings of the National Academy of Sciences; online November 6, 2006.

 

LV11.     Large-scale vector production (for future vector production for wide-spread vector manufacture)
Generation of a packaging cell line for prolonged large-scale production of high-titer HIV-1 based lentiviral vector
Ni Y, Sun S, Oparaocha I, Humeau L, Davis B, Cohen R, Binder G, Chang YN, Slepushkin V, Dropulic B.  Generation of a packaging cell line for prolonged large-scale production of high-titer HIV-1-based lentiviral vector   J Gene Med. 2005 Jun;7(6):818-34.

 

LV12.     A review of the history leading to the first lentiviral vector clinical trial
Regulatory Considerations for Novel Gene Therapy Products: A Review of the Process Leading to the First Clinical Lentiviral Vector
Manilla P, Rebello T, Afable C, Lu X, Slepushkin V, Humeau LM, Schonely K, Ni Y, Binder GK, Levine BL, MacGregor RR, June CH, Dropulic B.  Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector.   Hum Gene Ther. 2005 Jan;16(1):17-25.

 

LV13.      Inhibition of simian/human immunodeficiency virus
Inhibition of simian/human immunodeficiency virus replication in CD4+ T cells derived from lentiviral-transduced CD34+ hematopoietic cells
Braun SE, Wong FE, Connole M, Qiu G, Lee L, Gillis J, Lu X, Humeau L, Slepushkin V, Binder GK, Dropulic B, Johnson RP.  Inhibition of simian/human immunodeficiency virus replication in CD4+ T cells derived from lentiviral-transduced CD34+ hematopoietic cells.   Mol Ther. 2005 Dec;12(6):1157-67. Epub 2005 Sep 15.

 

LV14.     Method and efficiency of vector-mediated gene transfer to stem cells
ABC transporter inhibitors that are substrates enhance lentiviral vector transduction into primitive hematopoietic progenitor cells
Davis BM, Humeau L, Slepushkin V, Binder G, Korshalla L, Ni Y, Ogunjimi EO, Chang LF, Lu X, Dropulic B.  ABC transporter inhibitors that are substrates enhance lentiviral vector transduction into primitive hematopoietic progenitor cells.  Blood. 2004 Jul 15;104(2):364-73. Epub 2004 Apr 1.

LV15.     Method and efficiency of selection of vector-modified stem cells (to improve therapeutic efficacy of a stem cell-delivered gene product)
In Vivo Selection for Human and Murine Hematopoietic Cells Transduced with a Therapeutic MGMT Lentiviral Vector that Inhibits HIV Replication
Davis BM, Humeau L, Dropulic B.  In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication.   Mol Ther. 2004 Feb;9(2):160-72.

LV16.     Structure and safety features of VIRxSYS vector
Safe Two-plasmid Production for the First Clinical Lentivirus Vector that Achieves > 99% Transduction in Primary Cells Using a One-step Protocol
Lu X, Humeau L, Slepushkin V, Binder G, Yu Q, Slepushkina T, Chen Z, Merling R, Davis B, Chang YN, Dropulic B. Safe two-plasmid production for the first clinical lentivirus vector that achieves >99% transduction in primary cells using a one-step protocol.   J Gene Med. 2004 Sep;6(9):963-73.

 

LV17.     Vector-mediated anti-HIV gene transfer to T cells and anti-HIV efficacy
Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load
Humeau LM, Binder GK, Lu X, Slepushkin V, Merling R, Echeagaray P, Pereira M, Slepushkina T, Barnett S, Dropulic LK, Carroll R, Levine BL, June CH, Dropulic B.  Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load.   Mol Ther. 2004 Jun;9(6):902-13.

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LV18.     Lack of resistant HIV in response to anti-HIV vector therapy
Antisense-mediated inhibition of human immunodeficiency virus (HIV) replication because of an HIV type 1-based vector results in severely attenuated mutants incapable of developing resistance
Lu X, Yu Q, Binder GK, Chen Z, Slepushkina T, Rossi J, Dropulic B.  Antisense-mediated inhibition of human immunodeficiency virus (HIV) replication by use of an HIV type 1-based vector results in severely attenuated mutants incapable of developing resistance.   J Virol. 2004 Jul;78(13):7079-88. 

LV19.     Safety-related release testing for the HIV clinical trial
Release Testing of an HIV-1 Based Lentiviral Vector Lot and Transduced Cellular Product
Schonely K, Afable C, Slepushkin V, Lu X, Andre K, Boehmer J, Bengston K, Doub M, Cohen R, Berlinger D, Slepushkina T, Chen Z, Li Y, Binder G, Davis B, Humeau L, Dropulic B.  QC release testing of an HIV-1 based lentiviral vector lot and transduced cellular product.   2003. Bioproc. J. 2: 39-47. 

LV20.     Production of vector used in clinical trials
Large-scale Purification of a Lentiviral Vector by Size Exclusion Chromatography or Mustang Q Ion Exchange Capsule
Slepushkin V, Chang N, Cohen R, Gan Y, Jiang B, Deausen E, Berlinger D, Binder G, Andre K, Humeau L, Dropulic B.  Large-scale purification of a lentiviral vector by size exclusion chromatography or mustang Q ion exchange capsule. 2003. Bioprocessing J. 2: 89-95.

 

 

SM1.       A General Review on RNA Splicing and Disease

Poulos MG, Batra R, Charizanis K, Swanson MS.

Cold Spring Harb Perspect Biol. 2010 Nov 17.

 

SM2.       Correction of Inherited Skin Diseases with SMaRT^TM

Wally V, Brunner M, Lettner T, Wagner M, Koller U, Trost A, Murauer EM, Hainzl S, Hintner H, Bauer JW. Hum Mol Genet. 2010 Dec 1;19(23):4715-25.

 

SM3        Correction of Inherited Skin Diseases with SMaRT^TM 

         Functional Correction of Type VII Collagen Expression in Dystrophic Epidermolysis Bullosa.

Murauer EM, Gache Y, Gratz IK, Klausegger A, Muss W, Gruber C, Meneguzzi G, Hintner H, Bauer JW.  J Invest Dermatol. 2010 Aug 19.

 

Shababi M, Glascock J, Lorson C.

Hum Gene Ther. 2010 Aug 30.

 

SM5       Trans-splicing Efficiency and Restoration of SMN1 Function

Coady TH, Lorson CL.

J Neurosci. 2010 Jan 6;30(1):126-30.

 

SM6       Trans-splicing Efficiency and Restoration of SMN1 Function

Le Roy F, Charton K, Lorson CL, Richard I.

Trends Mol Med. 2009 Dec;15(12):580-91. Epub 2009 Nov 10. Review.

 

McManus CJ, Duff MO, Eipper-Mains J, Graveley BR.

Proc Natl Acad Sci U S A. 2010 Jul 20;107(29):12975-9.

 

Lorain S, Peccate C, Le Hir M, Garcia L.

PLoS One. 2010 May 28;5(5):e10894

SM9       Trans-splicing Into Albumin Transcripts
Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo
Novel trans-splicing strategy to “hijack” highly abundant transcripts such as “albumin pre-mRNA” and production of therapeutic proteins. Examples include human Factor VIII to correct bleeding disorder, human apoA-I protein to increase HDL; the good cholesterol and human papillomavirus type-16 E7 (HPV16-E7) single-chain antibody
Wang J, Mansfield SG, Cote CA, Du Jiang P, Ke W, Amar MA, Brewer Jr. BH, Remaley AT, McGarrity GJ, Garcia-Blanco MA and Puttaraju M. (2009) Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo. Molecular Therapy 17:343-351.

 

Rodriguez-Martin, T., Anthony, K., Garcia-Blanco, M.A., Mansfield, S.G., Anderton, B.H. and Gallo, J-M. (2009). Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome mediated RNA trans-splicing. Human Molecular Genetics. In press. 

 

 

 

SM13     Pre-mRNA Imaging Using RNA Trans-Splicing
A generalizable strategy for imaging pre-mRNA levels in living subjects using spliceosome-mediated RNA trans-splicing
Walls ZF, Puttaraju M, Temple GF, Gambhir SS. (2008) A generalizable strategy for imaging pre-mRNA levels in living subjects using spliceosome-mediated RNA trans-splicing. J Nucl Med. 49:1146-54. 

 

 

 

 

 

 

SM19     Correction of DNA Protein Kinase Deficiency Using RNA Trans-Splicing
Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery
Zayed H, Xia L, Yerich A, Yant SR, Kay MA, Puttaraju M, McGarrity GJ, Wiest DL, McIvor RS, Tolar J, Blazar BR. (2007) Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Mol Ther. 15:1273-9. 

SM20.    Alternative Splicing
Alternative splicing: therapeutic target and tool
Review article on alternative splicing and therapeutic targets. Main emphasis is on antisense oligonucleotides for modulating splicing and other alternative approaches to reprogram RNAs including SMaRTTM
Garcia-Blanco MA. (2006) Alternative splicing: therapeutic target and tool. Prog Mol Subcell Biol. 44:47-64.

 

SM21.    RNA Repair For Hemophilia A
RNA repair for hemophilia A
Review article on SMaRTTM, special focus on repair of hemophilia A mutations
Chao H, Walsh CE. (2006) RNA repair for hemophilia A. Expert Rev Mol Med. 8:1-8.

 

SM22.    Reprogramming of Tau Alternative Splicing
Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies
Rodriguez-Martin T, Garcia-Blanco MA, Mansfield SG, Grover AC, Hutton M, Yu Q, Zhou J, Anderton BH, Gallo JM. (2005) Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies. Proc Natl Acad Sci U S A. 102:15659-64.

 

SM23.    Spliceosome-Mediated RNA Trans-Splicing With rAAV
Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells
Use of recombinant adeno-associated virus (rAAV) vectors F508) in polarized human CFDexpressing CFTR PTMs to repair the major mutation ( airway epithelial cells and restoration of function
Liu X, Luo M, Zhang LN, Yan Z, Zak R, Ding W, Mansfield SG, Mitchell LG, Engelhardt JF. (2005) Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells. Hum Gene Ther. 16:1116-23.

 

SM24.    Alternative Splicing
Making antisense of splicing
Review article on alternative splicing and creation of abundant therapeutic targets for oligonucleotide-based molecular approaches for modulating various types of splicing events as well as other RNA repair/reprogram strategies
Garcia-Blanco MA. Making antisense of splicing. Curr Opin Mol Ther. (2005) 7:476-82.

 

SM25.    Spliceosome-mediated RNA Trans-Splicing
Spliceosome-mediated RNA trans-splicing
Review article on SMaRTTM, main focus is on repair of hemophilia A mutations
Yang Y, Walsh CE. (2005) Spliceosome-mediated RNA trans-splicing. Mol Ther. 12:1006-12.

 

SM26.    Gene Transfer-Mediated Pre-mRNA Segmental Trans-Splicing for Cancer Therapy
Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy
Nakayama K, Pergolizzi RG, Crystal RG. (2005). Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy. Cancer Res. 65:254-63.

 

SM27.    Reprogramming Gene Expression By Trans-Splicing
Gene therapy progress and prospects: reprogramming gene expression by trans-splicing
Review article on SMaRTTM – an update on recent developments and progress
Mitchell LG, McGarrity GJ. (2005) Gene therapy progress and prospects: reprogramming gene expression by trans-splicing. Gene Ther. 12:1477-85.

 

SM28.    RNA Repair Via RNA Trans-Splicing
RNA repair using spliceosome-mediated RNA trans-splicing
Review article describes cis-splicing, trans-splicing and different types of SMaRTTM confirmed and available for mRNA repair, potential advantageous and limitations of SMaRTTM compared to traditional gene therapy strategies
Mansfield SG, Chao H, Walsh CE. (2004). RNA repair using spliceosome-mediated RNA trans-splicing. Trends Mol Med. 10:263-8.

 

SM29.    Lentivral Vector For Correction of X-Linked Immunodeficiency in Mice
Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency
Ex vivo gene therapy with a lentiviral vector expressing PTM for correction of X-linked immunodeficiency mutations in mice
Tahara M, Pergolizzi RG, Kobayashi H, Krause A, Luettich K, Lesser ML, Crystal RG. (2004). Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency. Nat Med. 10: 835-841.

 

SM30.    Conventional Gene Replacement vs. SMaRTTM
Genetic medicine at the RNA level: modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing
Review article describes the differences between conventional gene replacement strategy vs. SMaRTTM to modify the genetic information to treat genetic and acquired disorders, the main advantageous and limitations etc   
Pergolizzi RG, Crystal RG. (2004) Genetic medicine at the RNA level: modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing. C R Biol.  327:695-709.

 

SM31.    Proof of Concept of SMaRT for Visualizing Gene Expression
Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing
First proof-of-concept demonstrating the use of SMaRTTM for visualizing gene expression
Bhaumik S, Walls Z, Puttaraju M, Mitchell LG, Gambhir SS. (2004). Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing. Proc. Natl. Acad. Sci. U S A. 101:8693-8698.

 

SM32.    Alternative Splicing
Alternative splicing in disease and therapy
Review article on alternative splicing and its effect in creating diversity, human disease and available therapies to repair genetic mutations including SMaRTTM
Garcia-Blanco MA, Baraniak AP, Lasda EL. (2004). Alternative splicing in disease and therapy. Nat Biotechnol. 22:535-46.

 

SM33.    Proof of Concept of Segmental Trans-Splicing in Mice
In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer
Proof-of-concept showing the production of functional cobratoxin in mice via co-delivery of two plasmids termed “segmental trans-splicing”
Pergolizzi RG, Ropper AE, Dragos R, Reid AC, Nakayama K, Tan Y, Ehteshami JR, Coleman SH, Silver RB, Hackett NR, Menez A, Crystal RG. (2003) In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer. Mol Ther. 8:999-1008.

 

SM34.    Correction of Pathogenic Mutations by SMaRTTM
Type XVII collagen gene mutations in junctional epidermolysis bullosa and prospects for gene therapy
Review article summarizes the results for correction of pathogenic mutations responsible for skin disease (epidermolysis bullosa) by SMaRTTM  
Bauer JW, Lanschuetzer C. (2003). Type XVII collagen gene mutations in junctional epidermolysis bullosa and prospects for gene therapy. Clin Exp Dermatol. 28:53-60.

 

SM35.    Use of SMaRTTM for Antibodies Production
In-cell generation of antibody single-chain Fv transcripts by targeted RNA trans-splicing
Use of SMaRTTM for antibodies production  
Schlesinger J, Arama D, Noy H, Dagash M, Belinky P, Gross G. (2003). In-cell generation of antibody single-chain Fv transcripts by targeted RNA trans-splicing. J Immunol Methods. 282:175-86.

 

SM36.    SMaRTTM Compared to "Spicing Endonuclease"
Mending the message
News and views on another form of mRNA repair using “splicing endonuclease” compared to SMaRTTM and ribozymes
Garcia-Blanco MA. (2003) Mending the message. Nat Biotechnol. 21:1448-9.

 

SM37.    Repair of Hemophilia A Mutations by SMaRTTM
Phenotype Correction of Hemophilia A Mice by Spliceosome-Mediated RNA Trans-Splicing
Repair of mutations responsible for bleeding disorder (hemophilia A) by SMaRTTM-PTMs leading to restoration of phenotype in mice  
Chao, H., Mansfield, S. G., Bartel, R., Hiriyanna, S., Mitchell, L. G., Garcia-Blanco, M. A., and Walsh, C. E. (2003). Phenotype Correction of Hemophilia A Mice by Spliceosome-Mediated RNA Trans-Splicing. Nature Medicine. 9:1015-9.

 

SM38.    Messenger RNA Reprogramming by SMaRTTM
Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing
Garcia-Blanco MA. (2003). Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing. J Clin Invest.112:474-80.

 

SM39.    Correction of Inherited Skin Diseases Using SMaRT^TM
Development of Spliceosome-Mediated RNA Trans-Splicing (SMaRT™) for the Correction of Inherited Skin Diseases
Development of SMaRTTM for correction of inherited skin diseases
Dallinger, G., Puttaraju, M., Mitchell, L. G., Yancey, K. B., Yee, C., Klausegger, A., Hintner, H., and Bauer, J. W. (2003).  Development of Spliceosome-Mediated RNA Trans-Splicing (SMaRT™) for the Correction of Inherited Skin Diseases.  Experimental Dermatology 12: 37-46.

 

SM40.    Re-Programming Gene Expression Using SMaRT^TM
Re-Programming Gene Expression Using Spliceosome-Mediated RNA Trans-Splicing (SMaRT™)
Review of current status of SMaRT™ technology
Otto, E., Temple, G. F., and McGarrity, G. J. (2003).  Re-Programming Gene Expression Using Spliceosome-Mediated RNA Trans-Splicing (SMaRT™). Current Drug Discovery. Pages 37-42.

 

SM41.    Repair by RNA Trans-Splicing
5’ Exon Replacement and Repair by Spliceosome-Mediated RNA Trans-Splicing
Mansfield, S. G., Hawkins-Clark, R., Puttaraju, M., Kole, J., Cohn, J. A., Mitchell, L. G., and Garcia-Blanco, M. A. (2003). 5’ Exon Replacement and Repair by Spliceosome-Mediated RNA Trans-Splicing. RNA.  9:1290-7.

 

SM42.    Emerging RNA Therapeutics Including SMaRT^TM
Emerging clinical applications of RNA.
Review article on emerging RNA therapeutics including SMaRTTM
Sullenger BA, Gilboa E. (2002) Emerging clinical applications of RNA. Nature. 418:252-258.

 

SM43.    Repair of F508 CFTR Mutation Using SMaRT^TM
Functional Restoration of CFTR Chloride Conductance in Human CF Epithelia by Spliceosome-Mediated RNA Trans-Splicing
Repair of the major F508 CFTR mutation in human cystic fibrosis airway cells and restoration ofD function using SMaRTTM
Liu, X., Jiang, Q., Mansfield, S. G., Puttaraju, M., Zhang, Y., Zhou, W., Garcia-Blanco, M. A., Mitchell, L. G., and Engelhardt, J. F. (2002).  Functional Restoration of CFTR Chloride Conductance in Human CF Epithelia by Spliceosome-Mediated RNA Trans-Splicing.  Nature Biotechnology 20: 47-52.

 

SM44.    Messenger RNA Repair Using SMaRT^TM
Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing
Puttaraju, M., DiPasquale J, Baker CC, Mitchell LG, Garcia-Blanco MA. (2001). Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing.  Mol Ther 4: 105-114.

 

SM45.    Development of SMaRTTM to Replace Part of Cancer Causing Sequence

Development of SMaRTTM to replace part of cancer causing sequence in mutant RET protooncogene

Kikumori T, Cote GJ, Gagel RF. (2001). Promiscuity of pre-mRNA spliceosome-mediated trans-splicing: a problem for gene therapy? Hum Gene Ther. 12:1429-41.

 

 

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